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As research into Angelman syndrome continues to move forward, many families are beginning to think about clinical trials and what taking part might mean for them.
We know this can feel exciting, but also a little overwhelming.
The aim of this page is to bring together trusted information that will help you better understand the clinical trial process, the language you may hear, and the questions you might want to ask. We hope these resources will help you feel informed and prepared, whatever stage of the journey you’re on.
This 30-minute presentation was delivered by Dr Allyson Berent, Chief Science Officer at FAST Global, during the FAST Global Conference in November 2025.
Dr Berent explains:
How clinical trials work what families should know before considering participation
the current Angelman syndrome clinical trial landscape what to expect over the coming years.
Presentation Slides:
Download the accompanying slides to follow along with the presentation or refer back to key information at your own pace.
Download: HERE
This community webinar, hosted by FAST and the Angelman Syndrome Association (ASA), features representatives from Ionis and Ultragenyx discussing Phase 3 clinical trials in Europe and the UK.
Topics include:
Roadmap to a Cure – Family Guide to Angelman Syndrome
Our Roadmap to a Cure Family Guide includes a dedicated section, “From Benchside to Bedside: Understanding Clinical Trials,” which explains:
Looking for information about Angelman syndrome clinical trials? FAST’s Clinical Trial Finder lets you explore studies using filters such as age, location and recruiting status.
Explore FAST’s Clinical Trial Finder
Study information can change, and search results do not confirm eligibility or availability. Always check the full study record on ClinicalTrials.gov and contact the study team to confirm participation requirements.
Clinical trials can sometimes change, pause or stop earlier than planned. Find out why this might happen, how families may hear updates, and where to turn for information and support.
When a clinical trial changes or ends
Taking part in a clinical trial can involve hope, commitment and uncertainty for the whole family. Understanding what might happen if a trial changes, pauses or stops early can help families feel more prepared.
Why might a trial pause or stop early?
Clinical trials may be paused or stopped for different reasons. These can include concerns about safety, findings suggesting a treatment is unlikely to deliver the benefit being studied, or practical, funding or business decisions.
A pause does not necessarily mean that a trial will end permanently, and a trial stopping does not automatically mean the treatment was unsafe. The reasons and implications need to be understood for each individual trial.
How and when might families hear news?
There may be uncertainty about when an update will be available and how much information it will contain. An initial announcement may come before the full results have been analysed, so some questions may take longer to answer.
For publicly listed companies, rules about sharing information that could affect their share price can influence the timing and way announcements are made. This may mean that families see news, whether encouraging or disappointing, through a public announcement, news report or social media before receiving a direct update.
We recognise how difficult this can be, especially for families taking part. Families deserve clear, timely and compassionate communication from the organisations running trials and their clinical teams. FAST UK is advocating for better communication about trial participation, updates and outcomes, and we hope to see positive changes in this area.
What happens if our family is taking part?
Any questions about your trial should be directed to the trial’s investigation team in the first instance. They should explain what any change means for you or your child, including arrangements for treatment, appointments, safety monitoring and follow-up care. Access to the trial treatment after a study ends is not guaranteed; your team can explain the arrangements for your particular trial.
Useful questions to ask include:
• Why has the trial been paused or stopped?
• What does this mean for treatment and upcoming appointments?
• Will any follow-up visits or safety checks be needed?
• Who should we contact with questions or concerns?
• When and how should we expect the next update?
If you hear news online and are unsure what it means for your family, contact your trial team. Do not change any treatment or care arrangements without speaking to them.
How FAST UK will support families
FAST UK will do everything we can to share accurate, up-to-date information as quickly as possible through our website and social media channels.
We may not receive advance notice of announcements or have all the answers immediately. We will be clear about what has been confirmed, what remains uncertain and where families can find further information.
If you have questions or would like support, you can email info@cureangelman.org.uk Our team will respond as soon as we can, help you find reliable information and signpost you to appropriate support.
For questions about your or your child’s treatment, safety or care within a trial, please contact your trial team directly.
Clinical trials often involve scientific and medical terms that may be unfamiliar. We’ve created this glossary to help explain some of the words and phrases you may come across when learning about Angelman syndrome research and clinical trials.
A person’s genetic makeup. There are five recognised genetic causes (genotypes) of Angelman syndrome:
Imprinting is a natural process where only one copy of a gene is active, depending on whether it was inherited from the mother or the father. In Angelman syndrome, the paternal copy of the UBE3A gene is naturally switched off (silenced) in brain cells, meaning only the maternal copy is normally active.
A procedure in which a needle is inserted into the lower back, between the bones of the spine, to collect cerebrospinal fluid (CSF), administer medication or carry out medical tests. It is sometimes referred to as a spinal tap.
A research study that follows people over time without giving an experimental treatment. It helps researchers understand how Angelman syndrome changes naturally and provides important information that can be used to design and measure future clinical trials.
The tests or assessments used to determine whether a treatment has made a difference. They are usually carried out before treatment begins to provide a baseline and are repeated afterwards to measure any changes.
A treatment that contains no active medicine. In some clinical trials, a placebo is used to compare against the study treatment so researchers can better understand whether the treatment is having an effect.
A pretend medical procedure used in some clinical trials so participants do not know whether they received the active treatment. This helps ensure the trial results are as fair and unbiased as possible.
UBE3A is the gene responsible for Angelman syndrome. Most people have two copies of this gene, one inherited from each parent. In the brain, however, only the copy inherited from the mother is normally active. Angelman syndrome occurs when the maternal copy is missing or not working properly and the paternal copy remains naturally switched off.
Disclaimer: The definitions above have been simplified to explain how these terms relate to Angelman syndrome. They are intended as a helpful guide for families and should not replace the full scientific or medical definitions used by researchers or healthcare professionals.
Understanding Angelman syndrome can feel overwhelming at times, whether you are newly diagnosed, supporting a loved one, explaining the condition to others or simply looking to learn more.
This section brings together a range of helpful, easy-to-access educational resources from across the Angelman syndrome community. From basic genetics information and family-friendly science guides to lived experience stories and research updates, these tools are designed to support families, carers, professionals and anyone wanting to better understand Angelman syndrome.
Our FAST Science Booklet for Families presents Angelman syndrome research in a simple, visual, and accessible way. This booklet helps families understand current studies, scientific approaches and what research means for the community. You can now explore the digital version as a flipbook.
Educational webinars on topics such as Augmentative and Alternative Communication (AAC), behavioral strategies, and anxiety management.
School friendly resources designed to help children and young people understand Angelman Syndrome in an age-appropriate, engaging way. Whether you’re a parent, teacher, teaching assistant, or young carer, these presentations are tailored to support learning and inclusion at every stage of education.
Sessions from various years, covering topics like behavioural and anxiety concerns in Angelman Syndrome.
Content from the 2016 symposium addressing specific genetic aspects related to Angelman Syndrome.
FAST provides a comprehensive, regularly updated overview of all therapeutic approaches in development for Angelman syndrome, from gene therapy and paternal gene activation to downstream pathway treatments, showing the full global pipeline of research progressing toward effective therapies.
A collection of resources designed to support healthcare professionals in managing Angelman syndrome. These materials include guidance on the diagnostic pathway and best practice care following diagnosis, helping clinicians provide informed, coordinated support for individuals and families.
Stay connected with FAST UK through our twice-yearly newsletter, bringing together research updates, community stories, fundraising highlights and ways to get involved.
Whether you’re a family member, supporter, healthcare professional or researcher, our newsletter is a chance to catch up on our work and the community helping us move closer to a cure for Angelman syndrome.
There are many disorders that will not be cured or treated in our lifetime, but Angelman Syndrome will not be one of them. at FAST UK